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- US Clinical Trials Registry
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Clinical Trials on Genetic Disease, X-Linked
Total 15138 results
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Zhongmou TherapeuticsRecruitingX-linked RetinoschisisChina
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Shanghai General Hospital, Shanghai Jiao Tong University...Innostellar BiotherapeuticsRecruitingX-linked RetinoschisisChina
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Bio Products LaboratoryCompletedCommon Variable Immunodeficiency | Primary Immune Deficiency Disorders | X-linked Agammaglobulinaemia | Hyperimmunoglobulin M SyndromeUnited States
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AO GENERIUMRecruitingMetabolic Diseases | Mucopolysaccharidosis Type IIRussian Federation
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JCR Pharmaceuticals Co., Ltd.RecruitingMucopolysaccharidosis IIUnited States, Germany, Spain, France, Brazil, Argentina, Turkey, United Kingdom, Poland, Italy
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Denali Therapeutics Inc.Enrolling by invitationMucopolysaccharidosis IIUnited States, Canada, Belgium, Czechia, Netherlands
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Denali Therapeutics Inc.Active, not recruitingMucopolysaccharidosis IIUnited States, Netherlands, Canada, United Kingdom
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JCR Pharmaceuticals Co., Ltd.Enrolling by invitationMucopolysaccharidosis IIUnited States
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JCR Pharmaceuticals Co., Ltd.Completed
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REGENXBIO Inc.CompletedMucopolysaccharidosis IIBrazil, United States, United Kingdom
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REGENXBIO Inc.Enrolling by invitation
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ArmaGen, IncCompletedMucopolysaccharidosis IIUnited States, Germany, Philippines
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Janssen Pharmaceutical K.K.RecruitingX-Linked Retinitis PigmentosaJapan
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Frontera TherapeuticsPeking Union Medical College Hospital; Eye & ENT Hospital of Fudan University; Henan Provincial People's HospitalRecruitingX-Linked Retinitis PigmentosaChina
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Janssen Pharmaceutical K.K.CompletedX-Linked Retinitis PigmentosaJapan
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Applied Genetic Technologies CorpCompletedX-linked RetinoschisisUnited States
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Yale UniversityNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)CompletedHypophosphatemic Rickets, X Linked DominantUnited States
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Stanford UniversityNational Heart, Lung, and Blood Institute (NHLBI); University of California... and other collaboratorsCompletedMuscular Dystrophy, DuchenneUnited States
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Mayo ClinicCompleted
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Baylor College of MedicineUniversity of California, San Diego; University of Alabama at Birmingham; Boston... and other collaboratorsCompletedPrader-Willi Syndrome | Rett Syndrome | Angelman Syndrome | Sleep ProblemsUnited States
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TakedaRecruiting
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TakedaAvailableHunter SyndromeUnited States, Australia, Mexico, Spain, United Kingdom
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Green Cross CorporationCompleted
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Hacettepe UniversityCompletedChewing Problem | Swallowing DisorderTurkey
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National Center for Research Resources (NCRR)University of California, San DiegoCompleted
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Atsena Therapeutics Inc.RecruitingX-linked RetinoschisisUnited States
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University of ManchesterManchester University NHS Foundation Trust; Great Ormond Street Hospital for... and other collaboratorsRecruiting
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GC Biopharma CorpRecruitingMucopolysaccharidosis II | Hunter SyndromeKorea, Republic of
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Denali Therapeutics Inc.RecruitingMucopolysaccharidosis IIUnited States, Spain, United Kingdom, Czechia, France, Canada, Italy, Belgium, Netherlands, Germany, Argentina, Sweden, Turkey, Brazil, Colombia
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Homology Medicines, IncWithdrawnMucopolysaccharidosis IIUnited States, Canada
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JCR Pharmaceuticals Co., Ltd.Active, not recruitingMucopolysaccharidosis IIBrazil
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ShireCompletedMucopolysaccharidosis IIUnited States, United Kingdom, Brazil, Germany
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AO GENERIUMCompletedA Multi-cohort Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy VolunteersMetabolic Diseases | Mucopolysaccharidosis Type IIRussian Federation
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GlaxoSmithKlineCompletedMucopolysaccharidosis IIJapan
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Green Cross CorporationUnknownMucopolysaccharidosis II
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Janssen Research & Development, LLCJanssen Research & Development, LLCActive, not recruitingX-Linked Retinitis PigmentosaUnited States, Canada, Israel, United Kingdom, Spain, Denmark, France, Belgium, Italy, Netherlands, Switzerland
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BiogenCompletedX-Linked Retinitis PigmentosaUnited States, United Kingdom
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4D Molecular TherapeuticsActive, not recruitingX-Linked Retinitis PigmentosaUnited States
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Applied Genetic Technologies CorpCompletedX-Linked Retinitis PigmentosaUnited States
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Beacon TherapeuticsActive, not recruitingX-Linked Retinitis PigmentosaUnited States
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Beacon TherapeuticsActive, not recruitingX-Linked Retinitis PigmentosaUnited States
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Beacon TherapeuticsEnrolling by invitationX-Linked Retinitis PigmentosaUnited States
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Uskudar UniversityRecruitingLesch-Nyhan SyndromeTurkey
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ShireCompletedHunter SyndromeUnited States, Spain, Canada, United Kingdom, Mexico, Australia, France
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Green Cross CorporationCompleted
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ShireCompleted
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Green Cross CorporationUnknownThe Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) PatientsHunter SyndromeKorea, Republic of
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Kevin FlaniganMuscular Dystrophy AssociationTerminated
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BioLeaders CorporationCompleted
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Summit TherapeuticsCompletedModified Diet Trial: A Study of SMT C1100 in Paediatric Patients With DMD Who Follow a Balanced DietMuscular Dystrophy, DuchenneUnited Kingdom